Drugs / Rilzabrutinib
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Wayrilz | — | 2025-12-22 | europa.eu ↗ |
| Approved | US (FDA) | WAYRILZ | — | 2025-08-29 | fda.gov ↗ |
Trials 21
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 16 trials | ||||||
| Phase 1 | NCT07853131 | Oct 2026 → Apr 2027 expected | impaired renal function disease | Sanofi | Not yet recruiting | No outcome recorded |
| Phase 1 | NCT07855250 | Oct → Nov 2026 expected | — | Sanofi | Recruiting | No outcome recorded |
| Phase 1 | NCT06342700 | Mar → Apr 2024 | autoimmune disease | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT04748926 | Apr → May 2021 | — | Principia Biopharma, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT06444191 | Jan → Apr 2021 | — | Principia Biopharma, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT06444204 | Nov 2020 → Mar 2021 | liver disorder | Principia Biopharma, a Sanofi Company | Completed | No outcome recorded |
| Phase 28 trials · 2 met primary · 2 missed | ||||||
| Phase 2 | NCT06984627 | Sep 2025 → Feb 2027 expected | Graves disease | Sanofi | Recruiting | No outcome recorded |
| Phase 2 | NCT06500702 | Dec 2024 → Dec 2026 expected | focal segmental glomerulosclerosis, minimal change disease | Sanofi | Recruiting | No outcome recorded |
| Phase 2 | NCT05104892 | Dec 2021 → Feb 2024 | asthma | Sanofi | Completed | Missed primary |
| Phase 2 | NCT05002777 | Dec 2021 → May 2024 overdue | autoimmune hemolytic anemia, warm type | Sanofi | Active not recruiting | No outcome recorded |
| Phase 2 | NCT05107115 | Nov 2021 → Jul 2023 | chronic urticaria | Sanofi | Completed | Met primary |
| Phase 2 | NCT05018806 | Sep 2021 → Jun 2023 | atopic eczema | Sanofi | Completed | Missed primary |
| Phase 2 | NCT04520451 | Aug 2020 → Oct 2024 | immunoglobulin G4-related sclerosing disease | Principia Biopharma, a Sanofi Company | Completed | No outcome recorded |
| Phase 2 | NCT03395210 | Mar 2018 → Jan 2023 | autoimmune thrombocytopenia | Principia Biopharma, a Sanofi Company | Completed | Met primary |
| Phase 37 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT07007962 | Oct 2025 → Nov 2027 expected | thrombocytopenia due to immune destruction | Sanofi | Recruiting | No outcome recorded |
| Phase 3 | NCT07216079 | Oct 2025 → Nov 2026 expected | autoimmune thrombocytopenia | Sanofi | Active not recruiting | No outcome recorded |
| Phase 3 | NCT07190196 | Sep 2025 → Jun 2028 expected | immunoglobulin G4-related sclerosing disease | Sanofi | Recruiting | No outcome recorded |
| Phase 3 | NCT07086976 | Aug 2025 → May 2028 expected | autoimmune hemolytic anemia, warm type | Sanofi | Recruiting | No outcome recorded |
| Phase 3 | NCT06975865 | Aug 2025 → Jul 2027 expected | sickle cell disease | Sanofi | Recruiting | No outcome recorded |
| Phase 3 | NCT04562766 | Dec 2020 → Jan 2025 | autoimmune thrombocytopenia | Principia Biopharma, a Sanofi Company | Completed | Met primary |
| Phase 3 | NCT03762265 | Jan 2019 → Jul 2021 | pemphigus | Principia Biopharma, a Sanofi Company | Terminated | Missed primary Stop: Business |
News releases announcing trial results or a regulatory action · 18
| Date | Issuer | Release |
|---|---|---|
| 2026-06-23 | Sanofi | Regulatory Press Release: Sanofi’s Wayrilz approved in Japan to treat immune thrombocytopenia sanofi.com ↗
Sanofi’s Wayrilz approved in Japan to treat immune thrombocytopenia |
| 2026-03-02 | Sanofi | Regulatory Press Release: Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease sanofi.com ↗
The Ministry of Health, Labour and Welfare (MHLW) in Japan has granted orphan drug designation to rilzabrutinib, a novel, oral, reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, for IgG4-related disease (IgG4-RD). |
| 2026-02-09 | Sanofi | Regulatory Press Release: Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia sanofi.com ↗ |
| 2025-12-23 | Sanofi | Regulatory Press Release: Sanofi’s Wayrilz approved in the EU as the first BTK inhibitor to treat immune thrombocytopenia sanofi.com ↗
The European Commission has approved Wayrilz (rilzabrutinib), a novel, oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. |
| 2025-10-17 | Sanofi | Regulatory Press Release: Sanofi’s Wayrilz recommended for EU approval by the CHMP to treat immune thrombocytopenia sanofi.com ↗
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Wayrilz (rilzabrutinib) as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. |
| 2025-08-29 | Sanofi | Regulatory Press Release: Sanofi’s Wayrilz approved in US as first BTK inhibitor for immune thrombocytopenia sanofi.com ↗
The US Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. |
| 2025-08-14 | Sanofi | Regulatory Press Release: Sanofi’s rilzabrutinib earns orphan designation in the EU for IgG4-related disease sanofi.com ↗
The European Medicines Agency has granted orphan designation to rilzabrutinib, a reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, for IgG4-related disease (IgG4-RD). |
| 2025-06-12 | Sanofi | Results Media Update: EULAR: rilzabrutinib data in IgG4-related disease show reduction in flares and key disease markers; earns Fast Track designation in the US sanofi.com ↗
New data from a phase 2 study showed that treatment with rilzabrutinib led to a considerable reduction in disease flares and other key disease markers, as well as glucocorticoid (GC) sparing, in patients with active IgG4-related disease (IgG4-RD). |
| 2025-06-03 | Sanofi | Regulatory Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease sanofi.com ↗
The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation, in sickle cell disease. |
| 2025-05-01 | Sanofi | Results Media Update: ATS: Sanofi showcases leadership and breadth of scientific innovation in chronic inflammatory respiratory conditions sanofi.com ↗
Results from a phase 2 study showing rilzabrutinib reduced loss of asthma control events in uncontrolled adult patients with moderate-to-severe asthma, as highlighted by a decrease in rescue medication use over 12 weeks. |
| 2025-04-03 | Sanofi | Regulatory Press Release: Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines sanofi.com ↗
The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, an investigational, novel, advanced, oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for two rare diseases, warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD). |
| 2024-12-07 | Sanofi | Results Press Release: ASH: rilzabrutinib demonstrated significant patient benefit in the first positive phase 3 study of a BTK inhibitor in ITP sanofi.com ↗
Platelet response was achieved in 65% (n=86) of patients receiving rilzabrutinib compared to 33% (n=23) of patients on placebo. |
| 2024-05-22 | Sanofi | Results Media Update: New results from rilzabrutinib phase 2 study show potential to be first advanced oral treatment for moderate-to-severe asthma sanofi.com ↗
Late-breaking data presented at ATS show that treatment with rilzabrutinib led to a numerical reduction in loss of asthma control events, improvement in symptoms and was well-tolerated with no new safety signals observed |
| 2024-04-23 | Sanofi | Results Press Release: Rilzabrutinib LUNA 3 phase 3 study met primary endpoint in immune thrombocytopenia sanofi.com ↗
Positive results from the LUNA 3 phase 3 study demonstrated that rilzabrutinib 400 mg twice daily orally achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP). |
| 2024-02-24 | Sanofi | Results Press Release: Phase 2 results demonstrate rilzabrutinib rapidly reduced itch severity and significantly improved disease activity in adults with chronic spontaneous urticaria sanofi.com ↗
Positive results from the Phase 2 study RILECSU showed that rilzabrutinib significantly improved itch, hives and urticaria in adults with moderate-to-severe chronic spontaneous urticaria (CSU), whose symptoms are not adequately controlled by H1 antihistamines. |
| 2022-04-14 | Sanofi | Results Press Release: Positive Phase 1/2 study results of rilzabrutinib in people with immune thrombocytopenia published in The New England Journal of Medicine sanofi.com ↗
Results published today in the New England Journal of Medicine showed that treatment with rilzabrutinib resulted in a rapid and durable increase in platelet count in patients with heavily pretreated immune thrombocytopenia (ITP) |
| 2021-09-09 | Sanofi | Results Sanofi provides update on Phase 3 study evaluating rilzabrutinib for the treatment of pemphigus sanofi.com ↗
The Phase 3 PEGASUS trial evaluating rilzabrutinib to treat pemphigus, a rare autoimmune skin condition, did not meet its primary or key secondary endpoints. |
| 2020-11-18 | Sanofi | Regulatory Press Release: Rilzabrutinib granted FDA Fast Track Designation for treatment of immune thrombocytopenia sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to the oral investigational Bruton’s tyrosine kinase (BTK) inhibitor, rilzabrutinib, which has the potential to be the first BTK inhibitor for the treatment of immune thrombocytopenia (ITP). |
All press releases naming this drug 18 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 22 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rilzabrutinib | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07216079 ↗ |
| Known as | PRN1008 | “A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor Rilzabrutinib (PRN1008) in Moderate to Severe Pemphigus” NCT03762265 ↗4“An Open-Label, Phase 1 Study to Evaluate the Effect of Mild and Moderate Hepatic Impairment on the Single-Dose Pharmacokinetics of Rilzabrutinib (PRN1008)” NCT06444204 ↗ “Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)” NCT04562766 ↗ “Pharmacokinetics (PK) of Rilzabrutinib (PRN1008) in Healthy Japanese and Caucasian Subjects” NCT06444191 ↗ “rilzabrutinib (SAR444671 [Formerly PRN1008])” NCT04748926 ↗ |
| Known as | PRN1008/SAR444671 | “An Open Label, Two-Arm Study to Evaluate the Effect of Rilzabrutinib (PRN1008/SAR444671) on Safety and Disease Activity in ParticipantsWith IgG4-Related Disease” NCT04520451 ↗ |
| Known as | SAR444671 | “rilzabrutinib (SAR444671 [Formerly PRN1008])” NCT04748926 ↗ |
| Action | Inhibit | “Oral BTK Inhibitor Rilzabrutinib (PRN1008)” NCT03762265 ↗ |
| Route | Oral | “We used intrapatient dose escalation of oral rilzabrutinib over a period of 24 weeks” PMID 35417637 ↗ Apr 2022 |
| Target | BTK | “Oral BTK Inhibitor Rilzabrutinib (PRN1008)” NCT03762265 ↗ |